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Orphan Drugs: Is There A Relationship Between Disease Prevalence And Price ?

September 12, 2014

Much discussion has recently centered on the pricing of orphan drugs. Why are the prices of orphan drugs and of other new medications (especially for oncology indications) so high?  Is there a relationship between the price of an orphan drug and the prevalence of the rare disease it treats?

A report, from Thomson Reuters, “Lysosomal Storage Disease: An example of a lucrative ultra-orphan market”, by Ulrike Jahnke, reviews the LSD market and the advantages of ultra-orphan therapies. One interesting topic addressed in the report is the question of whether or not there is a relationship between the prevalence of a rare disease and the corresponding price of the disease’s approved drug. Jahnke evaluates this question by looking at the current LSD market. The article proposes that there is

“…. an inverse correlation between drug price and the prevalence of disease …. with the treatment being more expensive the rarer the disease.”

Jahnke addresses this theory by looking at particular LSDs, estimated number of patients affected, and the price of the treatment/patient/year. The following chart (created from data in the report) identifies the following data fields:

•   Drug Name/Type of Therapy

•   Indication

•   Sponsor Company

•   Approximate # patients affected

•   Price/patient/year.

Correlation Between Prevalence And Price

Drug Name/Type of Therapy Indication Sponsor Company Approx. # of Patients Price/Patient/Per Year
Naglazyme/ ERT Injection Maroteaux-Lamy Syndrome (MPS VI) BioMarin 1,100 $375,000
Elaprase/ ERT Injection Hunter Syndrome (MPS II) Shire 2,000 $375,000
Vimizim/ ERT Injection Morquio A (MPS IV A) BioMarin 3,000 $380,000
Aldurazyme/ ERT Injection Hurler (MPS I) Genzyme (Sanofi)/ BioMarin 3,000 – 4,000 $200,000
Fabrazyme/ ERT Injection Fabry Disease Genzyme (Sanofi) 5,000 – 10,000 $200,000
Replagal/ ERT Injection Fabry Disease Shire 5,000 – 10,000 $150,000 – $200,000
Myozyme/ ERT Injection Pompe Disease Genzyme (Sanofi) < 10,000 $300,000
Cerezyme/ ERT Injection Gaucher Disease Genzyme (Sanofi) < 10,000 $200,000
Vpriv/ ERT Injection Gaucher Disease Shire < 10,000 $170,000
Elelyso/ ERT Injection Gaucher Disease Pfizer/ Protalix Biotherapeutics < 10,000 $150,000
Cerdelga ** / Oral Pill Gaucher Disease Genzyme (Sanofi) <10,000 $310,250

*   ERT = Enzyme Replacement Therapy

** Cerdelga data not from report.


Please Note: “3D Bar Graph Meeting” by “The Gold Guys Blog” [CC-BY-SA-2.0] via Wikimedia Commons..

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